Agenda Details

Session Details

10:15 AM – 12:00 PM - Room 408 AB

Co-Chair

Kathleen M. Kirby. , Barnstable, MA

10:16 AM – 10:25 AM - Room 408 AB

Natural History Studies - Introduction

Kathleen M. Kirby, BS. Viridian, Barnstable, MA

10:25 AM – 10:37 AM - Room 408 AB

How to design an NH study that regulators and clinicians support

Adam Shaywitz, MD, PhD. BridgeBio Pharma, Palo Alto, CA

10:37 AM – 10:49 AM - Room 408 AB

Designing a development plan for a robust control data source

John Balser, PhD. Veristat LLC, MA

10:49 AM – 11:01 AM - Room 408 AB

Listening to the public/patients/caregivers

Cara O'Neill, MD. Cure Sanfilippo Foundation, SC

11:01 AM – 11:13 AM - Room 408 AB

Running NH studies in a robust and verifiable way

Anne Ruggles. Pariser, MD. Alltrna, Bethesda, MD

11:13 AM – 11:48 AM - Room 408 AB

Panelist

Cara O'Neill, MD. Cure Sanfilippo Foundation, SC

11:13 AM – 11:48 AM - Room 408 AB

Panelist

Kristen Pappas. , CA

11:13 AM – 11:48 AM - Room 408 AB

Panelist

Anne Ruggles. Pariser, MD. Alltrna, Bethesda, MD

11:13 AM – 11:48 AM - Room 408 AB

Panelist

Adam Shaywitz, MD PhD. BridgeBio Pharma, Palo Alto, CA

11:13 AM – 11:48 AM - Room 408 AB

Panelist

John Balser. , MA

11:48 AM – 12:00 PM - Room 408 AB

Key take-aways; Future directions

Samantha Parker. Innoskel, Biot, France